Practical DMD Course 03. Pathophysiology and Natural History of DMD

This module provides a comprehensive and up-to-date overview of the pathophysiology and natural history of Duchenne muscular dystrophy (DMD), addressing the molecular and cellular mechanisms underlying the disease and their clinical implications. Through a practical and multidisciplinary approach, the course explores the fundamental role of dystrophin, alterations in muscle regeneration, inflammation, fibrosis, and the functional progression experienced by patients. In addition, the module reviews the main clinical signs and early manifestations of DMD, highlighting the importance of early diagnosis and the recognition of both motor and non-motor symptoms. The course also analyzes the factors influencing clinical evolution, including genetics, corticosteroid treatment, and current standards of care, as well as the multisystemic impact of the disease. Designed for healthcare professionals involved in the management of patients with DMD, this content provides the essential foundations for understanding the disease from both pathophysiological and clinical perspectives, facilitating improved identification, follow-up, and therapeutic management of these patients.